Last reviewed · How we verify
Buphenyl
At a glance
| Generic name | Buphenyl |
|---|---|
| Also known as | Sodium Phenylbutyrate, NaPBA |
| Sponsor | Amgen |
| Target | Aldose reductase, Histone deacetylase 1, Histone deacetylase 2 |
| Modality | Small molecule |
| Therapeutic area | Metabolic |
| Phase | FDA-approved |
Approved indications
- Amyotrophic lateral sclerosis
- Citrullinemia
- Congenital hyperammonemia, type I
- Deficiency of carbamylphosphate synthetase (CPS)
- Disorder of the urea cycle metabolism
- Ornithine Carbamyltransferase Deficiency
Common side effects
- Acidosis
- Amenorrhea/Menstrual dysfunction
- Hypoalbuminemia
- Anemia
- Alkalosis
- Hyperchloremia
- Increased alkaline phosphatase
- Hypophosphatemia
- Decreased appetite
- Increased liver transaminases
- Leukopenia
- Leukocytosis
Serious adverse events
- Aplastic anemia
- Pancreatitis
- Peptic ulcer disease
- Rectal bleeding
- Arrhythmia
- Renal tubular acidosis
- Neurotoxicity
- Syncope
Key clinical trials
- PROFIL Study to Investigate the Effect of GPB on NfL Levels in Patients With Corticobasal Syndrome (CBS) (PHASE2)
- Na-Phenylbutyrate VAscular Trial (EARLY_PHASE1)
- Phenylbutyrate for Monogenetic Developmental and Epileptic Encephalopathy (EARLY_PHASE1)
- A Clinical Study of Glycerol Phenylbutyrate in Chinese Patients With Urea Cycle Disorders
- Efficacy and Safety of the Treatment of Pyruvate Dehydrogenase Deficiency Patients With Glycerol Phenylbutyrate (RAVICTI) (PHASE2)
- Phenylbutyrate in Proteinuric Nephropathies (PHASE2, PHASE3)
- Study of Sodium Phenylbutyrate (ACER-001) for the Treatment of Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD) (PHASE2)
- PBA Use for Treatment of ATF6-/- Patients (EARLY_PHASE1)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Buphenyl CI brief — competitive landscape report
- Buphenyl updates RSS · CI watch RSS
- Amgen portfolio CI